Mai ElMallah | Scholars@Duke

Mai ElMallah
Associate Professor of Pediatrics

Our laboratory focuses on the control of breathing and pulmonary mechanics in murine models of several genetic diseases. We are currently focuased on ALS mouse models and the Pompe disease mouse model. We examine the impact of gene therapy and neuromodulation on respiratory insufficiency that results from neuromuscular weakness. As a clinician scientist, my goal is to bring therapy from the bench to the bedside and enhance our research at the bench through observations at the bedside.

Current Appointments & Affiliations

Associate Professor of Pediatrics, Pediatrics, Pulmonary and Sleep Medicine, Pediatrics 2018

Contact Information

mai.elmallah@duke.edu

Background
Education, Training, & Certifications
- Pediatric Pulmonary Fellowship, Pediatrics, University of Florida College of Medicine 2010 - 2012
- Residency, Pediatrics, Baylor College of Medicine 2002 - 2005
- Junior House Officer, Queen Elizabeth Hospital, England 2001 - 2002
- M.B.B.Ch., Queens University Belfast (N. Ireland) 2001
Duke Appointment History
- Instructor in the Department of Pediatrics, Pediatrics, Pulmonary and Sleep Medicine, Pediatrics 2017 - 2018
Expertise
Subject Headings
Research
Selected Grants
- Mental Health Screening in Duke Pediatric CF Patients awarded by Cystic Fibrosis Foundation 2018 - 2021
- Evolving High Potency AAV Vectors for Neuromuscular Genome Editing awarded by National Institutes of Health 2018 - 2021
- VX16-809-120 awarded by Vertex Pharmaceuticals Inc. 2018 - 2021
- A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease awarded by National Institutes of Health 2017 - 2020
- Sav005-04 awarded by Savara Inc. 2017 - 2019
- Cystic Fibrosis Foundation awarded by Cystic Fibrosis Foundation 2018 - 2019
- Gene Therapy for Upper Airway and Respiratory Insufficiency in an ALS Mouse Model awarded by National Institutes of Health 2016 - 2019
- Parker B. Francis Jo Rae Wright Award for Scientific Excellence awarded by Parker B. Francis Foundation 2018 - 2019
- Novel Approaches for Correcting Respiratory Insufficiency in Pompe Disease awarded by National Institutes of Health 2014 - 2019
Publications & Artistic Works
Selected Publications
- Academic Articles
  - McCall, Angela L., and Mai K. ElMallah. “Macroglossia, Motor Neuron Pathology, and Airway Malacia Contribute to Respiratory Insufficiency in Pompe Disease: A Commentary on Molecular Pathways and Respiratory Involvement in Lysosomal Storage Diseases..” Int J Mol Sci 20, no. 3 (February 11, 2019). https://doi.org/10.3390/ijms20030751.
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  - Keeler, Allison M., Marina Zieger, Sophia H. Todeasa, Angela L. McCall, Jennifer C. Gifford, Samantha Birsak, Sourav R. Choudhury, Barry J. Byrne, Miguel Sena-Esteves, and Mai K. ElMallah. “Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease..” Hum Gene Ther 30, no. 1 (January 2019): 57–68. https://doi.org/10.1089/hum.2018.016.
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  - Borel, Florie, Huaming Sun, Marina Zieger, Andrew Cox, Brynn Cardozo, Weiying Li, Gabriella Oliveira, et al. “Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema..” Proc Natl Acad Sci U S A 115, no. 11 (March 13, 2018): 2788–93. https://doi.org/10.1073/pnas.1713689115.
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  - McCall, Angela L., Jeffrey Salemi, Preeti Bhanap, Laura M. Strickland, and Mai K. Elmallah. “The impact of Pompe disease on smooth muscle: a review..” J Smooth Muscle Res 54, no. 0 (2018): 100–118. https://doi.org/10.1540/jsmr.54.100.
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  - “Erratum for “Pediatric Obstructive Sleep Apnea in High-Risk Populations: Clinical Implications”.” Pediatric Annals 46, no. 11 (November 1, 2017): e436–e436. https://doi.org/10.3928/19382359-20171018-05.
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  - “Erratum for “Pediatric Obstructive Sleep Apnea and Asthma: Clinical Implications”.” Pediatric Annals 46, no. 11 (November 1, 2017): e436–e436. https://doi.org/10.3928/19382359-20171018-04.
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  - ElMallah, Mai, Evan Bailey, Michelle Trivedi, Ted Kremer, and Lawrence M. Rhein. “Pediatric Obstructive Sleep Apnea in High-Risk Populations: Clinical Implications..” Pediatr Ann 46, no. 9 (September 1, 2017): e336–39. https://doi.org/10.3928/19382359-20170815-01.
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  - Trivedi, Michelle, Mai ElMallah, Evan Bailey, Ted Kremer, and Lawrence M. Rhein. “Pediatric Obstructive Sleep Apnea and Asthma: Clinical Implications..” Pediatr Ann 46, no. 9 (September 1, 2017): e332–35. https://doi.org/10.3928/19382359-20170815-03.
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  - Keeler, A. M., M. K. ElMallah, and T. R. Flotte. “Gene Therapy 2017: Progress and Future Directions..” Clin Transl Sci 10, no. 4 (July 2017): 242–48. https://doi.org/10.1111/cts.12466.
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  - Keeler, Allison M., Donghai Liu, Marina Zieger, Lang Xiong, Jeffrey Salemi, Karl Bellvé, Barry J. Byrne, David D. Fuller, Ronghua ZhuGe, and Mai K. ElMallah. “Airway smooth muscle dysfunction in Pompe (Gaa-/- ) mice..” Am J Physiol Lung Cell Mol Physiol 312, no. 6 (June 1, 2017): L873–81. https://doi.org/10.1152/ajplung.00568.2016.
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  - Stoica, Lorelei, Allison M. Keeler, Lang Xiong, Michael Kalfopoulos, Kaitlyn Desrochers, Robert H. Brown, Miguel Sena-Esteves, Terence R. Flotte, and Mai K. ElMallah. “Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model..” Am J Respir Cell Mol Biol 56, no. 3 (March 2017): 405–8. https://doi.org/10.1165/rcmb.2016-0258LE.
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  - Turner, S. M., M. K. ElMallah, A. K. Hoyt, J. J. Greer, and D. D. Fuller. “Ampakine CX717 potentiates intermittent hypoxia-induced hypoglossal long-term facilitation..” J Neurophysiol 116, no. 3 (September 1, 2016): 1232–38. https://doi.org/10.1152/jn.00210.2016.
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  - Turner, Sara M. F., Aaron K. Hoyt, Mai K. ElMallah, Darin J. Falk, Barry J. Byrne, and David D. Fuller. “Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice..” Respir Physiol Neurobiol 227 (June 15, 2016): 48–55. https://doi.org/10.1016/j.resp.2016.02.007.
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  - Loring, Heather S., Mai K. ElMallah, and Terence R. Flotte. “Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans..” Hum Gene Ther Methods 27, no. 2 (April 2016): 49–58. https://doi.org/10.1089/hgtb.2015.150.
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  - Stoica, Lorelei, Sophia H. Todeasa, Gabriela Toro Cabrera, Johnny S. Salameh, Mai K. ElMallah, Christian Mueller, Robert H. Brown, and Miguel Sena-Esteves. “Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model..” Ann Neurol 79, no. 4 (April 2016): 687–700. https://doi.org/10.1002/ana.24618.
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  - ElMallah, Mai K., David A. Stanley, Kun-Ze Lee, Sara M. F. Turner, Kristi A. Streeter, David M. Baekey, and David D. Fuller. “Power spectral analysis of hypoglossal nerve activity during intermittent hypoxia-induced long-term facilitation in mice..” J Neurophysiol 115, no. 3 (March 2016): 1372–80. https://doi.org/10.1152/jn.00479.2015.
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  - Borel, Florie, Gwladys Gernoux, Brynn Cardozo, Jake P. Metterville, Gabriela C. Toro Cabrera, Lina Song, Qin Su, et al. “Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates..” Hum Gene Ther 27, no. 1 (January 2016): 19–31. https://doi.org/10.1089/hum.2015.122.
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  - ElMallah, Mai K., Silvia Pagliardini, Sara M. Turner, Anthony J. Cerreta, Darin J. Falk, Barry J. Byrne, John J. Greer, and David D. Fuller. “Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines..” Am J Respir Cell Mol Biol 53, no. 3 (September 2015): 326–35. https://doi.org/10.1165/rcmb.2014-0374OC.
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  - Bacewicz, Aleksandra, Wei Wang, Judy Ashouri, and Mai K. ElMallah. “Children with Chronic Lung Disease: Facilitating Smoking Cessation for their Caregivers..” J Community Health 40, no. 3 (June 2015): 409–13. https://doi.org/10.1007/s10900-014-9947-2.
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  - Halawa, M., M. N. Abu-Hasan, and M. K. ElMallah. “Hereditary mucoepithelial dysplasia and severe respiratory distress.” Respiratory Medicine Case Reports 15 (April 8, 2015): 27–29. https://doi.org/10.1016/j.rmcr.2015.03.004.
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  - Falk, Darin J., Adrian Gary Todd, Sooyeon Lee, Meghan S. Soustek, Mai K. ElMallah, David D. Fuller, Lucia Notterpek, and Barry J. Byrne. “Peripheral nerve and neuromuscular junction pathology in Pompe disease..” Hum Mol Genet 24, no. 3 (February 1, 2015): 625–36. https://doi.org/10.1093/hmg/ddu476.
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  - Abu-Hasan, Mutasim, Mai Elmallah, Dan Neal, and James Brookes. “Salivary Amylase Level in Bronchoalveolar Fluid as a Marker of Chronic Pulmonary Aspiration in Children..” Pediatr Allergy Immunol Pulmonol 27, no. 3 (September 1, 2014): 115–19. https://doi.org/10.1089/ped.2014.0348.
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  - Elmallah, Mai K., Darin J. Falk, Sushrusha Nayak, Roland A. Federico, Milapjit S. Sandhu, Amy Poirier, Barry J. Byrne, and David D. Fuller. “Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice..” Mol Ther 22, no. 4 (April 2014): 702–12. https://doi.org/10.1038/mt.2013.282.
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  - Fuller, David D., Mai K. ElMallah, Barbara K. Smith, Manuela Corti, Lee Ann Lawson, Darin J. Falk, and Barry J. Byrne. “The respiratory neuromuscular system in Pompe disease..” Respir Physiol Neurobiol 189, no. 2 (November 1, 2013): 241–49. https://doi.org/10.1016/j.resp.2013.06.007.
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  - Falk, Darin J., Cathryn S. Mah, Meghan S. Soustek, Kun-Ze Lee, Mai K. Elmallah, Denise A. Cloutier, David D. Fuller, and Barry J. Byrne. “Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease..” Mol Ther 21, no. 9 (September 2013): 1661–67. https://doi.org/10.1038/mt.2013.96.
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  - Elmallah, M. K., and L. Hendeles. “Delivery of medications by metered dose inhaler through a chamber/mask to young children with asthma.” Pediatric, Allergy, Immunology, and Pulmonology 25, no. 4 (December 1, 2012): 236–39. https://doi.org/10.1089/ped.2012.0197.
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  - ElMallah, Mai K., Darin J. Falk, Michael A. Lane, Thomas J. Conlon, Kun-Ze Lee, Nadeem I. Shafi, Paul J. Reier, Barry J. Byrne, and David D. Fuller. “Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9..” Hum Gene Ther Methods 23, no. 2 (April 2012): 148–56. https://doi.org/10.1089/hgtb.2012.009.
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  - Elmallah, Mai K., Yasmeen Khan, Guenther Hochhaus, Jonathan J. Shuster, and Leslie Hendeles. “Systemic exposure to fluticasone MDI delivered through antistatic chambers..” J Allergy Clin Immunol 128, no. 5 (November 2011). https://doi.org/10.1016/j.jaci.2011.06.014.
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  - ElMallah, Mai K., Sreekala Prabhakaran, and Sarah E. Chesrown. “Plastic bronchitis: resolution after heart transplantation..” Pediatr Pulmonol 46, no. 8 (August 2011): 824–25. https://doi.org/10.1002/ppul.21432.
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  - Byrne, Barry J., Darin J. Falk, Christina A. Pacak, Sushrusha Nayak, Roland W. Herzog, Melissa E. Elder, Shelley W. Collins, et al. “Pompe disease gene therapy..” Hum Mol Genet 20, no. R1 (April 15, 2011): R61–68. https://doi.org/10.1093/hmg/ddr174.
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  - Lee, Kun-Ze, Kai Qiu, Milapjit S. Sandhu, Mai K. Elmallah, Darin J. Falk, Michael A. Lane, Paul J. Reier, Barry J. Byrne, and David D. Fuller. “Hypoglossal neuropathology and respiratory activity in pompe mice..” Front Physiol 2 (2011). https://doi.org/10.3389/fphys.2011.00031.
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  - El Mallah, M. K., A. J. Sands, F. A. Casey, B. G. Craig, and H. C. Mulholland. “Transcatheter occlusion of the patent ductus arteriosus: a comparison of two devices..” Ir J Med Sci 171, no. 3 (July 2002): 151–54.
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  - El Mallah, M. K., U. Chakravarthy, and P. M. Hart. “Amblyopia: is visual loss permanent?.” Br J Ophthalmol 84, no. 9 (September 2000): 952–56. https://doi.org/10.1136/bjo.84.9.952.
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- Book Sections
  - Kalfopoulos, Michael, Kaitlyn Wetmore, and Mai K. ElMallah. “Pathophysiology of Alpha-1 Antitrypsin Lung Disease.,” 1639:9–19, 2017. https://doi.org/10.1007/978-1-4939-7163-3_2.
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  - Wang, W., A. Bacewicz, J. Ashouri, R. K. Elmallah, and M. K. ElMallah. “Secondhand smoke exposure in children with chronic lung disease.” In Nicotine Dependence, Smoking Cessation and Effects of Secondhand Smoke, 87–121, 2015.
- Conference Papers
  - Keeler, Allison M., Marina Zieger, Carson Semple, Ali Veinbachs, Guangping Gao, Robert H. Brown, Christian Mueller, and Mai K. ElMallah. “Respiratory Directed Gene Therapy to Silence SOD1 Prolongs Survival in the SOD1 ALS Mouse.” In Molecular Therapy, 27:422–422. CELL PRESS, 2019.
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  - Zieger, Marina, Allison M. Keeler, Terence R. Flotte, and Mai K. ElMallah. “AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency..” In J Inherit Metab Dis, 2019. https://doi.org/10.1002/jimd.12101.
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  - King, S., J. A. Sank, J. Hildebran, B. McLurkin, and M. K. ElMallah. “A RESPIRATORY THERAPIST-DRIVEN QUALITY IMPROVEMENT INITIATIVE IN THE EFFECTIVE MANAGEMENT OF AIRWAY CLEARANCE FOR THE PEDIATRIC PATIENT WITH CYSTIC FIBROSIS.” In Pediatric Pulmonology, 53:381–82. WILEY, 2018.
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  - Lind, Lori A., Katherine A. Johnson, Angela L. McCall, Christian Mueller, Robert H. Brown, Teresa E. Lever, Mai K. Elmallah, and Nicole L. Nichols. “Impact of Intralingual AAVrh10-miRSOD1 Injection on Respiratory Function in SOD1(G93A) Mice.” In Faseb Journal, Vol. 32. FEDERATION AMER SOC EXP BIOL, 2018.
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  - ElMallah, M. K., A. Keeler, M. Zieger, F. Borel, V. Semple, A. Veinbachs, R. Brown, and C. Mueller. “Respiratory Directed Gene Therapy Prolongs Survival In An Als Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 197. AMER THORACIC SOC, 2018.
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  - Borel, Florie, Huaming Sun, Mai K. Elmallah, Andrew Cox, Brynn Cardozo, Weiying Li, Andrew M. Hoffman, Terence R. Flotte, and Christian Mueller. “Editing out Five SERPINA1 Paralogs to Create a New Mouse Model of Genetic Emphysema.” In Molecular Therapy, 25:117–117. CELL PRESS, 2017.
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  - Keeler-Klunk, Allison M., Marina Zieger, Samantha Bircsak, Sourav Choudhury, Barry J. Byrne, Miguel Sena-Esteves, and Mai K. ElMallah. “Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe Disease.” In Molecular Therapy, 25:28–29. CELL PRESS, 2017.
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  - Keeler, A. M., M. Zieger, D. Liu, J. Salemi, R. ZhuGe, and M. K. ElMallah. “Respiratory And Airway Smooth Muscle Pathology In A Pompe Disease Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 195. AMER THORACIC SOC, 2017.
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  - Lu, P., D. Liu, K. Bellve, K. Fogarty, M. K. ElMallah, L. Lifshitz, and R. ZhuGe. “Bitter Tastant 1,10-Phenanthroline Activates Tas2r-Gustducin Signaling To Relax Airways, Resulting In A Decrease In Airway Resistance In Vivo.” In American Journal of Respiratory and Critical Care Medicine, Vol. 195. AMER THORACIC SOC, 2017.
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  - Borel, Florie, Brynn Cardozo, Andrew Cox, Weiying Li, Andrew Hoffman, Mai ElMallah, and Christian Mueller. “167. Genome Editing to Generate the First Mouse Model of Alpha-One Antitrypsin Deficiency, the Leading Cause of Genetic COPD.” In Molecular Therapy, 24:S65–S65. Elsevier BV, 2016. https://doi.org/10.1016/s1525-0016(16)32976-8.
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  - Turner, Sara, Mai Elmallah, Aaron Hoyt, John Greer, and David Fuller. “Pretreatment with Ampakine CX717 Enhances Long-Term Facilitation (LTF) of Inspiratory Hypoglossal (XII) Bursting, But Only When Initial Burst Output is Low.” In Faseb Journal, Vol. 30. FEDERATION AMER SOC EXP BIOL, 2016.
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  - Keeler, A. M., K. V. Desroshers, L. Xiong, and M. K. ElMallah. “Exercise Induced Respiratory Insufficiency In A Murine Model Of Very Long-Chain Acyl-Coa Dehydrogenase Deficiency.” In American Journal of Respiratory and Critical Care Medicine, Vol. 193. AMER THORACIC SOC, 2016.
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  - Stoica, L., L. Xiong, M. Kalfopoulos, K. V. Desroshers, and M. K. ElMallah. “Characterization Of Pulmonary Mechanics Of The Sod1g93a Als Mouse Model.” In American Journal of Respiratory and Critical Care Medicine, Vol. 193. AMER THORACIC SOC, 2016.
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  - ElMallah, Mai, David Stanley, and David Fuller. “Hypoglossal long term facilitation shifts the power spectral density to higher frequencies.” In Faseb Journal, Vol. 29. FEDERATION AMER SOC EXP BIOL, 2015.
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  - Turner, Sara, Mai ElMallah, Darin Falk, Barry Byrne, and David Fuller. “Preferential Neuropathology in Respiratory-Related Motor Nuclei in Pompe Mice.” In Faseb Journal, Vol. 29. FEDERATION AMER SOC EXP BIOL, 2015.
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  - Fuller, David, Mai Kamal Elmallah, Heather Ross, Anthony Cerreta, Manuela Corti, Barb Smith, Amy Poirier, Ronald Mandel, and Barry Byrne. “Selective knockout of acid alpha-glucosidase GAA gene expression in the medulla alters breathing.” In Faseb Journal, Vol. 28. FEDERATION AMER SOC EXP BIOL, 2014.
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  - ElMallah, M. K., S. Pagliardini, S. M. Turner, J. J. Greer, B. J. Byrne, and D. D. Fuller. “Ampakines Enhance Respiratory Motor Output In A Murine Model Of Pompe Disease.” In American Journal of Respiratory and Critical Care Medicine, Vol. 189. AMER THORACIC SOC, 2014.
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  - Falk, D. J., A. G. Todd, M. S. Soustek, D. C. Cloutier, M. ElMallah, D. D. Fuller, and B. J. Byrne. “P.17.9 AAV9 vector encoding hGAA improves synaptic pathology of the neuromuscular junction in Pompe disease.” In Neuromuscular Disorders, 23:829–829. Elsevier BV, 2013. https://doi.org/10.1016/j.nmd.2013.06.659.
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  - ElMallah, Mai Kamal, Milap S. Sandhu, Kun-Ze Lee, David M. Baekey, and David D. Fuller. “Apnea Alters the Pattern of Intermittent Hypoxia-Induced Long Term Facilitation in Anesthetized Mice.” In Faseb Journal, Vol. 27. FEDERATION AMER SOC EXP BIOL, 2013.
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  - Sandhu, Milap Singh, Mai K. Elmallah, Michael A. Lane, Paul J. Reier, John J. Greer, and David D. Fuller. “Ampakines induce a persistent increase in phrenic motor output in rats with cervical spinal cord injury.” In Faseb Journal, Vol. 27. FEDERATION AMER SOC EXP BIOL, 2013.
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  - Fuller, David, Mai Elmallah, Darin Falk, Lee Ann Lawson, A Daniel Martin, Barbara Smith, and Barry Byrne. “The importance of targeting the central nervous system for treatment of respiratory insufficiency in Pompe disease.” In Molecular Genetics and Metabolism, 108:S41–S41. Elsevier BV, 2013. https://doi.org/10.1016/j.ymgme.2012.11.089.
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  - Chakravarthy, U., M. El Mallah, and P. M. Hart. “Amblyopia: Is visual loss permanent..” In Investigative Ophthalmology & Visual Science, 41:S705–S705, 2000.
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Education, Training, & Certifications

Duke Appointment History

Subject Headings

Selected Grants

Selected Publications

Academic Articles

Book Sections

Conference Papers